PTC Therapeutics to Discuss Launch Plans for Emflaza™ (Deflazacort) during Community Call on Monday, May 8th at 3:00 PM EDT

PTC Therapeutics has released a letter to the community and will host a conference call for the Duchenne community to discuss the launch plans for EMFLAZA™ (deflazacort) on Monday, May 8, 2017 at 3:00 PM EDT (call-in details are below).

On a public call to investors earlier this morning, PTC announced that the price for EMFLAZA in the U.S. will start at $35,000 for a patient weighing 25kg (approximately 55lbs), and would fluctuate based on a patient’s dose and weight.

PPMD is anxious to learn more details about how they arrived at this pricing structure, as well as access plans, on today’s community call.

Call-in Details:

PTC Therapeutics, Inc. will host a conference call for the Duchenne community to discuss the launch plans for Emflaza™ (deflazacort) on Monday, May 8, 2017 at 3:00 pm (ET). The call can be accessed by dialing (877) 303-9216 (domestic) five minutes prior to the start of the call and providing the passcode 12438417.

UPDATE 5/8 - 12:30 PM: PTC has requested that questions be submitted in advance of the 3 PM EDT community call. If you have questions you would like PPMD to submit to PTC on your behalf, please email info@parentprojectmd.org (subject line: Emflaza call).

PTC's Letter to the Duchenne Community:

Dear Duchenne Families,

Today we shared our plans for the launch of EMFLAZA™ (deflazacort), the first FDA-approved corticosteroid for Duchenne muscular dystrophy patients age 5 and older, regardless of the genetic mutation. We are excited to launch EMFLAZA in the U.S., and we will begin making the product commercially available to Duchenne patients and families in the upcoming weeks. We have a team in place with extensive experience, including a dedicated field force to support Healthcare professionals and the patient community.

For nearly 20 years, PTC has been working to understand how to investigate Duchenne muscular dystrophy and to identify therapies that fundamentally change the lives of patients living with this disorder. The launch of EMFLAZA is another step toward achieving this goal. The approval of EMFLAZA for all Duchenne patients regardless of genetic mutation provides us with the opportunity to work with the whole Duchenne community to help standards of care. We will do this through awareness initiatives to enable diagnosis and access to treatment and specialty care.

We know some families are anxious about the affordability and access to EMFLAZA. In response, we have set a goal to provide EMFLAZA to all eligible Duchenne patients, regardless of insurance status or type, and have developed a process so that patients have seamless access to the appropriate service. We have a trained team of case managers at EMFLAZACares™ who will work with Duchenne patients every step of the way to help them get EMFLAZA with minimal difficulty and expense.

We appreciate that prior to FDA approval, access to deflazacort had been difficult and many patients were unable to get the drug. Our team at PTC is ready to deliver a consistent and reliable supply of EMFLAZA and has high quality assurance measures in place to oversee and manage production and inventory.

We are proud of our almost two decade involvement with the Duchenne community, and we are looking forward to taking this next step in our journey with you. From all of us at PTC, we thank you for your partnership in helping us bring this important therapy to Duchenne patients in the U.S.